Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain

Neuroscience

Volumn 138, Issue 2, 2006, Pages 501-510

  Broekman, M L D ^a,b   Comer, L A ^a   Hyman, B T ^a   Sena Esteves, M ^a,c  

^a MASSACHUSETTS GENERAL HOSPITAL   (United States)

^b UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^c Molecular Neurogenetics Unit   (United States)

Author keywords

AAV8; Adeno associated virus; Brain; Gene delivery; Neonatal; Serotypes

Indexed keywords

BETA ACTIN; GREEN FLUORESCENT PROTEIN; PARVOVIRUS VECTOR;

AAV1 GENE; AAV2 GENE; AAV8 GENE; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BRAIN; BRAIN CORTEX; CELL LINE; CEREBELLUM; FEMALE; GENE EXPRESSION; GENE STRUCTURE; GENE TARGETING; GENE VECTOR; HIPPOCAMPUS; LATERAL BRAIN VENTRICLE; LONG TERMINAL REPEAT; MALE; MOUSE; NONHUMAN; OLFACTORY BULB; PERINATAL PERIOD; PRIORITY JOURNAL; PROTEIN EXPRESSION; PURKINJE CELL; SEROTYPE; SIGNAL TRANSDUCTION; VIRUS CAPSID;

ANIMALS; ANIMALS, NEWBORN; BASE SEQUENCE; BRAIN; CAPSID; CELL LINE; CEREBELLUM; CEREBRAL CORTEX; DEPENDOVIRUS; DNA PRIMERS; GENETIC VECTORS; HIPPOCAMPUS; HUMANS; KIDNEY; MICE; MICE, INBRED C57BL; OLFACTORY BULB; SEROTYPING; TRANSFECTION;

EID: 32844456598     PISSN: 03064522     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.neuroscience.2005.11.057     Document Type: Article

References (51)

1. J. Altman, S.A. Bayer Migration and distribution of two populations of hippocampal granule cell precursors during the perinatal and postnatal periods J Comp Neurol 301 1990 365 381
2. J. Altman, S.A. Bayer Mosaic organization of the hippocampal neuroepithelium and the multiple germinal sources of dentate granule cells J Comp Neurol 301 1990 325 342
3. K.S. Bankiewicz, J.L. Eberling, M. Kohutnicka, W. Jagust, P. Pivirotto, J. Bringas, J. Cunningham, T.F. Budinger, J. Harvey-White Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach Exp Neurol 164 2000 2 14
4. J.S. Bartlett, R.J. Samulski, T.J. McCown Selective and rapid uptake of adeno-associated virus type 2 in brain Hum Gene Ther 9 1998 1181 1186
5. C. Burger, O.S. Gorbatyuk, M.J. Velardo, C.S. Peden, P. Williams, S. Zolotukhin, P.J. Reier, R.J. Mandel, N. Muzyczka Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system Mol Ther 10 2004 302 317
6. C. Burger, F.N. Nguyen, J. Deng, R.J. Mandel Systemic mannitol-induced hyperosmolality amplifies rAAV2-mediated striatal transduction to a greater extent than local co-infusion Mol Ther 11 2005 327 331
7. B.L. Davidson, C.S. Stein, J.A. Heth, I. Martins, R.M. Kotin, T.A. Derksen, J. Zabner, A. Ghodsi, J.A. Chiorini Recombinant adeno-associated virus type 2, 4, and 5 vectors transduction of variant cell types and regions in the mammalian central nervous system Proc Natl Acad Sci U S A 97 2000 3428 3432
8. G. Di Pasquale, B.L. Davidson, C.S. Stein, I. Martins, D. Scudiero, A. Monks, J.A. Chiorini Identification of PDGFR as a receptor for AAV-5 transduction Nat Med 9 2003 1306 1312
9. H. Fu, J. Muenzer, R.J. Samulski, G. Breese, J. Sifford, X. Zeng, D.M. McCarty Self-complementary adeno-associated virus serotype 2 vector global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain Mol Ther 8 2003 911 917
10. G. Gao, L.H. Vandenberghe, M.R. Alvira, Y. Lu, R. Calcedo, X. Zhou, J.M. Wilson Clades of adeno-associated viruses are widely disseminated in human tissues J Virol 78 2004 6381 6388
11. G.P. Gao, M.R. Alvira, L. Wang, R. Calcedo, J. Johnston, J.M. Wilson Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy Proc Natl Acad Sci U S A 99 2002 11854 11859
12. P. Hadaczek, H. Mirek, J. Bringas, J. Cunningham, K. Bankiewicz Basic fibroblast growth factor enhances transduction, distribution, and axonal transport of adeno-associated virus type 2 vector in rat brain Hum Gene Ther 15 2004 469 479
13. B. Hauck, W. Xiao Characterization of tissue tropism determinants of adeno-associated virus type 1 J Virol 77 2003 2768 2774
14. B. Hauck, W. Zhao, K. High, W. Xiao Intracellular viral processing, not single-stranded DNA accumulation, is crucial for recombinant adeno-associated virus transduction J Virol 78 2004 13678 13686
15. D.M. Hoover, J. Lubkowski DNAWorks an automated method for designing oligonucleotides for PCR-based gene synthesis Nucleic Acids Res 30 2002 e43
16. B.T. Hyman, G.W. Van Hoesen, L.J. Kromer, A.R. Damasio Perforant pathway changes and the memory impairment of Alzheimer's disease Ann Neurol 20 1986 472 481
17. M. Jacobson Developmental neuroscience 3rd ed. 1991 Plenum Press New York
18. M.G. Kaplitt, P. Leone, R.J. Samulski, X. Xiao, D.W. Pfaff, K.L. O'Malley, M.J. During Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain Nat Genet 8 1994 148 154
19. M. Klugmann, C. Wymond Symes, C.B. Leichtlein, B.K. Klaussner, J. Dunning, D. Fong, D. Young, M.J. During AAV-mediated hippocampal expression of short and long Homer 1 proteins differentially affect cognition and seizure activity in adult rats Mol Cell Neurosci 28 2005 347 360
20. M. Klugmann, C.B. Leichtlein, C.W. Symes, B.C. Klaussner, A.I. Brooks, D. Young, M.J. During A novel role of circadian transcription factor DBP in hippocampal plasticity Mol Cell Neurosci Oct 26 2005 [Epub ahead of print] PMID: 16257226
21. W.D. Lo, G. Qu, T.J. Sferra, R. Clark, R. Chen, P.R. Johnson Adeno-associated virus-mediated gene transfer to the brain duration and modulation of expression Hum Gene Ther 10 1999 201 213
22. M.Y. Mastakov, K. Baer, R.M. Kotin, M.J. During Recombinant adeno-associated virus serotypes 2- and 5-mediated gene transfer in the mammalian brain quantitative analysis of heparin co-infusion Mol Ther 5 2002 371 380
23. M.Y. Mastakov, K. Baer, R. Xu, H. Fitzsimons, M.J. During Combined injection of rAAV with mannitol enhances gene expression in the rat brain Mol Ther 3 2001 225 232
24. R. Matalon, S. Surendran, P.L. Rady, M.J. Quast, G.A. Campbell, K.M. Matalon, S.K. Tyring, J. Wei, C.S. Peden, E.L. Ezell, N. Muzyczka, R.J. Mandel Adeno-associated virus-mediated aspartoacylase gene transfer to the brain of knockout mouse for canavan disease Mol Ther 7 2003 580 587
25. C.H. Miao, R.O. Snyder, D.B. Schowalter, G.A. Patijn, B. Donahue, B. Winther, M.A. Kay The kinetics of rAAV integration in the liver Nat Genet 19 1998 13 15
26. H. Nakai, Y. Iwaki, M.A. Kay, L.B. Couto Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver J Virol 73 1999 5438 5447
27. H. Nakai, S.R. Yant, T.A. Storm, S. Fuess, L. Meuse, M.A. Kay Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo J Virol 75 2001 6969 6976
28. H. Nakai, E. Montini, S. Fuess, T.A. Storm, M. Grompe, M.A. Kay AAV serotype 2 vectors preferentially integrate into active genes in mice Nat Genet 34 2003 297 302
29. H. Nakai, S. Fuess, T.A. Storm, S. Muramatsu, Y. Nara, M.A. Kay Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice J Virol 79 2005 214 224
30. J.B. Nguyen, R. Sanchez-Pernaute, J. Cunningham, K.S. Bankiewicz Convection-enhanced delivery of AAV-2 combined with heparin increases TK gene transfer in the rat brain Neuroreport 12 2001 1961 1964
31. A. Novak, C. Guo, W. Yang, A. Nagy, C.G. Lobe Z/EG, a double reporter mouse line that expresses enhanced green fluorescent protein upon Cre-mediated excision Genesis 28 2000 147 155
32. M. Okabe, M. Ikawa, K. Kominami, T. Nakanishi, Y. Nishimune 'Green mice' as a source of ubiquitous green cells FEBS Lett 407 1997 313 319
33. M.A. Passini, D.J. Watson, C.H. Vite, D.J. Landsburg, A.L. Feigenbaum, J.H. Wolfe Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-deficient mice J Virol 77 2003 7034 7040
34. M.A. Passini, J.H. Wolfe Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector J Virol 75 2001 12382 12392
35. J.C. Paterna, J. Feldon, H. Bueler Transduction profiles of recombinant adeno-associated virus vectors derived from serotypes 2 and 5 in the nigrostriatal system of rats J Virol 78 2004 6808 6817
36. C.S. Peden, C. Burger, N. Muzyczka, R.J. Mandel Circulating anti-wild-type adeno-associated virus type 2 (AAV2) antibodies inhibit recombinant AAV2 (rAAV2)-mediated, but not rAAV5-mediated, gene transfer in the brain J Virol 78 2004 6344 6359
37. K. Qing, C. Mah, J. Hansen, S. Zhou, V. Dwarki, A. Srivastava Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2 Nat Med 5 1999 71 77
38. J.E. Rabinowitz, F. Rolling, C. Li, H. Conrath, W. Xiao, X. Xiao, R.J. Samulski Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity J Virol 76 2002 791 801
39. M.R. Rosenfeld, I. Bergman, L. Schramm, J.A. Griffin, M.G. Kaplitt, P.I. Meneses Adeno-associated viral vector gene transfer into leptomeningeal xenografts J Neurooncol 34 1997 139 144
40. L.M. Sanftner, B.M. Suzuki, M.M. Doroudchi, L. Feng, A. McClelland, J.R. Forsayeth, J. Cunningham Striatal delivery of rAAV-hAADC to rats with preexisting immunity to AAV Mol Ther 9 2004 403 409
41. M. Sena-Esteves, J.C. Tebbets, S. Steffens, T. Crombleholme, A.W. Flake Optimized large-scale production of high titer lentivirus vector pseudotypes J Virol Methods 122 2004 131 139
42. C. Summerford, J.S. Bartlett, R.J. Samulski AlphaVbeta5 integrin a co-receptor for adeno-associated virus type 2 infection Nat Med 5 1999 78 82
43. C. Summerford, R.J. Samulski Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions J Virol 72 1998 1438 1445
44. C.E. Thomas, T.A. Storm, Z. Huang, M.A. Kay Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors J Virol 78 2004 3110 3122
45. M.R. Veldwijk, J. Topaly, S. Laufs, U.R. Hengge, F. Wenz, W.J. Zeller, S. Fruehauf Development and optimization of a real-time quantitative PCR-based method for the titration of AAV-2 vector stocks Mol Ther 6 2002 272 278
46. C. Wang, C.M. Wang, K.R. Clark, T.J. Sferra Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain Gene Ther 10 2003 1528 1534
47. Z. Wang, T. Zhu, C. Qiao, L. Zhou, B. Wang, J. Zhang, C. Chen, J. Li, X. Xiao Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart Nat Biotechnol 23 2005 321 328
48. D.J. Watson, M.A. Passini, J.H. Wolfe Transduction of the choroid plexus and ependyma in neonatal mouse brain by vesicular stomatitis virus glycoprotein-pseudotyped lentivirus and adeno-associated virus type 5 vectors Hum Gene Ther 16 2005 49 56
49. M.J. West, L. Slomianka, H.J. Gundersen Unbiased stereological estimation of the total number of neurons in the subdivisions of the rat hippocampus using the optical fractionator Anat Rec 231 1991 482 497
50. R. Xu, C.G. Janson, M. Mastakov, P. Lawlor, D. Young, A. Mouravlev, H. Fitzsimons, K.L. Choi, H. Ma, M. Dragunow, P. Leone, Q. Chen, B. Dicker, M.J. During Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes Gene Ther 8 2001 1323 1332
51. S. Zolotukhin, M. Potter, I. Zolotukhin, Y. Sakai, S. Loiler, T.J. Fraites Jr, V.A. Chiodo, T. Phillipsberg, N. Muzyczka, W.W. Hauswirth, T.R. Flotte, B.J. Byrne, R.O. Snyder Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors Methods 28 2002 158 167